2014-05-27T10:15:56-05:00 2013-12-31 231518888 MAILLIE LLP PO BOX 3068 WEST CHESTER PA 193813068 990 2013-01-01 522122720 FRIEDREICH'S ATAXIA RESEARCH ALLIANCE FRIE 4848796160 PO BOX 1537 SPRINGFIELD VA 22151 JENNIFER FARMER EXECUTIVE DIRECTOR 4848796160 2014-05-14 1 EDWARD FRONCZKOWSKI CPA P01259092 6106964353 2013 2015-11-30 17:44:51Z JENNIFER FARMER 533 W UWCHLAN AVENUE DOWNINGTOWN PA 19335 5226124 0 X WWW.CUREFA.ORG X 1998 VA TO TREAT AND CURE FRIEDREICH'S ATAXIA BY ADVANCING RESEARCH, AWARENESS AND PARTNERSHIPS. 16 15 6 350 0 0 3174652 3852500 0 0 21555 59864 127578 75480 3323785 3987844 2673157 2806246 0 0 420293 451977 0 0 158339 167762 211229 3261212 3469452 62573 518392 3032470 3688078 264360 381693 2768110 3306385 X TO MARSHAL AND FOCUS THE RESOURCES AND RELATIONSHIPS NEEDED TO CURE FA BY RAISING FUNDS FOR RESEARCH, PROMOTING PUBLIC AWARENESS AND ALIGNING SCIENTISTS, PATIENTS, CLINICIANS, GOV'T AGENCIES AND PHARMACEUTICAL COMPANIES DEDICATED TO CURING FA AND RELATED DISORDERS. 0 0 2992504 2806246 RESEARCH & GRANT PROGRAM: FARA'S COMMITMENT TO FUNDING HIGH QUALITY RESEARCH PROJECTS THAT MOVE US CLOSER TO EFFECTIVE TREATMENTS CONTINUED AT AN ACCELERATED PACE IN 2013. IN TOTAL, FARA PROVIDED >$2.8M IN BASIC AND TRANSLATIONAL AND CLINICAL RESEARCH GRANTS, SCIENTIFIC CONFERENCE SUPPORT AND CLINICAL RESEARCH INFRASTRUCTURE FUNDING. OVER THE PAST YEAR, >26 NEW GRANT APPLICATIONS WERE SUBMITTED AND AFTER RIGOROUS PEER REVIEW, 13 WERE DETERMINED MERITORIOUS AND AWARDED FUNDING IN 2013 AND EARLY 2014. IN ADDITION, BASED ON EXCELLENT PROGRESS, CONTINUATION FUNDING WAS PROVIDED FOR 15 PROJECTS, FOR A TOTAL OF 28 RESEARCH GRANTS AWARDED. FARA ALSO AWARDED TWO CONFERENCE GRANTS TO SUPPORT SCIENTIFIC MEETINGS WITH CONTENT FOCUSED ON FRIEDREICH'S ATAXIA. THESE FUNDED PROJECTS ADDRESS NUMEROUS IMPORTANT RESEARCH QUESTIONS THAT DIRECTLY TARGET FARA'S STRATEGIC RESEARCH INITIATIVES, INCLUDING DRUG DISCOVERY, PRE-CLINICAL AND CLINICAL STUDIES OF LEAD CANDIDATES IN THE TREATMENT PIPELINE, ADVANCES IN GENE AND CELL THERAPY APPROACHES, ADVANCES IN DIFFERENTIATION OF INDUCED PLURIPOTENT STEM (IPS) CELL LINES FROM FA PATIENTS INTO SENSORY NEURONS AND CARDIOMYOCYTES, DEVELOPMENT OF NEW ANIMAL MODELS, CARDIAC RESEARCH AND EVALUATION OF NOVEL BIOMARKERS. SEVERAL ARE CO-FUNDED WITH OUR FRIEDREICH'S ATAXIA ADVOCACY GROUP PARTNERS; THIS COLLABORATIVE APPROACH BOTH INCREASES THE NUMBER OF AVAILABLE RESEARCH DOLLARS AND HELPS TO MINIMIZE DUPLICATION OF EFFORT. OF NOTE, FA SHARES SIMILAR SYMPTOMS AND DISEASE MECHANISMS WITH OTHER DISEASES, BOTH RARE AND COMMON. RESEARCH INTO FA IS PROVIDING INSIGHTS AND ADVANCES IN OTHER DISEASES SUCH AS MITOCHONDRIAL DISEASES, MUSCULAR DYSTROPHIES, DIABETES, AND CARDIOMYOPATHY. A SAMPLE OF 2013 FARA FUNDED GRANTS FOLLOWS BELOW: - NEW INVESTIGATOR AWARD: DR. JONATHAN JONES, UNIVERSITY MIGUEL HERNANDEZDR. JONES WAS THE RECIPIENT OF FARA'S NEW INVESTIGATOR AWARD FOR HIS WORK EXAMINING THE NEUROPROTECTIVE EFFECT OF BONE MARROW-DERIVED STEM CELLS IN A FRIEDREICH'S ATAXIA MOUSE MODEL. THIS IS A NEW THERAPEUTIC APPROACH UTILIZING ADVANCES IN STEM CELLS. STEM CELLS ARE AN INCREASINGLY POPULAR SOURCE FOR CELL THERAPY IN A VARIETY OF DISEASES. DR. JONES' PREVIOUS WORKS HAS PROVEN THAT BONE MARROW STEM CELLS ARE CAPABLE OF SAFELY INTEGRATING INTO THE NERVOUS TISSUE AND INDUCE A TROPHIC EFFECT, PROTECTING THE NEURONS IN NEURODEGENERATIVE MODELS SUCH AS A CEREBELLAR ATAXIA MODEL (JONES J ET AL 2010) AND A MOTORNEURON DEGENERATIVE MODEL (PASTOR D ET AL 2011), AS WELL AS IN HUMAN ALS PATIENTS (BLANQUER ET AL 2012). HOWEVER, IN ORDER TO CONSIDER THE USE OF STEM CELLS FOR THE TREATMENT OF FRIEDREICH'S ATAXIA (FA), IT IS NECESSARY TO STUDY THE FEASIBILITY, SAFETY AND TROPHIC EFFICACY OF THIS APPROACH IN THE LAB. OVERALL, THE RESULTS WILL HELP HIM UNDERSTAND IF AND HOW STEM CELLS PROTECT NEURONS IN FA, AND POSSIBLY DRIVE US TO A POTENTIAL THERAPEUTIC APPROACH FOR THE DISEASE. - KEITH MICHAEL ANDRUS MEMORIAL AWARD (FOR CARDIAC RESEARCH)DR. MARK PAYNE, INDIANA UNIVERSITY & DR. MATTHEW HIRSHEY, DUKE UNIVERSITYTHIS PROJECT IS A COLLABORATIVE EFFORT BETWEEN DR. MARK PAYNE, MD, AT THE INDIANA UNIVERSITY SCHOOL OF MEDICINE, AND DR. MATT HIRSCHEY, PHD, AT DUKE UNIVERSITY SCHOOL OF MEDICINE, TO UNDERSTAND THE BIOCHEMICAL BASIS OF HEART FAILURE IN FA. BOTH HAVE INDEPENDENTLY MADE RECENT NOVEL FINDINGS THAT MAY REPRESENT CRITICAL EVENTS IN THE MITOCHONDRIAL DYSFUNCTION THAT UNDERLIES THE FATAL CARDIOMYOPATHY OF FA: THE HIRSCHEY LAB HAS MADE THE FUNDAMENTAL FINDING THAT MITOCHONDRIAL PROTEINS CAN BE MODIFIED ('ACETYLATED') AND BECOME LESS ACTIVE, AND THAT A SPECIFIC ENZYME ('SIRT3') IS RESPONSIBLE FOR RESTORING THEIR ACTIVITY TO NORMAL. THE PAYNE LAB HAS MADE THE DISEASE-RELATED FINDING THAT MITOCHONDRIAL PROTEINS FROM THE HEART IN FA ARE HEAVILY MODIFIED ('ACETYLATED'), AND THIS MAY REDUCE THEIR ABILITY TO USE NORMAL FUELS (FAT) FOR ENERGY, THUS CAUSING HEART FAILURE. THEY PROPOSE TO COMBINE THE STRENGTHS OF BOTH LABS TO EXPLORE AND DETERMINE THE MECHANISM OF MITOCHONDRIAL DYSFUNCTION UNDERLYING HEART FAILURE IN FA. IF CORRECT, THESE FINDINGS WOULD IDENTIFY AN IMPORTANT MECHANISM UNDERLYING MITOCHONDRIAL DYSFUNCTION IN FA, AND WOULD EXPLAIN WHY FA PATIENTS DEVELOP A FATAL HYPERTROPHIC CARDIOMYOPATHY. FURTHERMORE, THESE FINDINGS WOULD IDENTIFY A NEW TREATMENT STRATEGY FOR FA PATIENTS BY PHARMACOLOGICALLY TARGETING PROTEIN ACETYLATION, RESULTING IN NEW AND INNOVATIVE APPROACHES FOR FA TREATMENT.- KYLE BRYANT TRANSLATIONAL RESEARCH AWARD: DR. MIRIAM CNOP, BRUSSELS, BELGIUMTHE AIM OF THIS PRECLINICAL PROJECT, FROM DR. MIRIAM CNOP, IS TO EVALUATE THE POTENTIAL OF INCRETIN ANALOGS TO INDUCE FRATAXIN AND PREVENT OR IMPROVE NEURODEGENERATION AND DIABETES IN THE KIKO MOUSE MODEL. IF SUCCESSFUL, SHE FORESEES THAT THESE FINDINGS CAN BE RAPIDLY TRANSLATED INTO A CLINICAL TRIAL OF INCRETIN ANALOGS IN FA PATIENTS, BECAUSE THE DRUGS ARE COMMERCIALLY AVAILABLE FOR THE TREATMENT OF DIABETES.- BRONYA J. KEATS INTERNATIONAL RESEARCH AWARD: DRS.MARTIN DELATYCKI AND KIMBERLY LIN, MELBOURNE, AUSTRALIA AND PHILADELPHIA, PATHE LARGE GAPS IN OUR KNOWLEDGE REGARDING THE NATURE AND CAUSES OF THE HEART INVOLVEMENT IN FRIEDREICH ATAXIA (FRDA) LIMIT OUR ABILITY TO INVESTIGATE POTENTIAL TREATMENTS IN THIS CONDITION. INCREASED THICKNESS OF THE HEART WALLS, A COMMON FEATURE IN FRDA, IS A PREDICTOR OF SYMPTOMATIC HEART DISEASE AND PREMATURE MORTALITY. BASED ON THIS INFORMATION ALONE, A REDUCTION IN WALL THICKNESS HAS BEEN CONSIDERED TO BE A VALID CLINICAL ENDPOINT IN SOME CLINICAL TRIALS. IT IS SUSPECTED THAT THE INCREASE IN WALL THICKNESS INVOLVES BOTH LOSS OF HEART MUSCLE CELLS AND AN INCREASE IN SCAR TISSUE. IT IS CRITICAL WE FURTHER OUR UNDERSTANDING OF THE MECHANISMS THAT CONTRIBUTE TO INCREASED WALL THICKNESS IN FRDA, AS THIS WILL HELP DETERMINE IF REDUCTION IN WALL THICKNESS IS A VALID OUTCOME MEASURE. A NEW CMR TECHNIQUE HAS SHOWN PROMISE IN DETECTING EARLY SCARRING BUT HAS NOT YET BEEN STUDIED IN FRDA. IN THIS COLLABORATIVE STUDY BETWEEN THE MELBOURNE AND PHILADELPHIA CCRN SITES WE PROPOSE TO PERFORM CMR ON 105 INDIVIDUALS WITH FRDA TO DETERMINE THE FREQUENCY AND EXTENT OF SCARRING IN THE HEART MUSCLE WALL. AN IMPORTANT PART OF THIS STUDY IS THE INVESTIGATION OF THE RELATIONSHIP OF HEART CHANGES IN FRDA WITH ONE OF THE BODY'S NATURAL HORMONAL SYSTEMS, ALREADY TO KNOWN TO PLAY A ROLE IN OTHER TYPES OF HEART DISEASE. A SECOND IMPORTANT PART OF THE STUDY WILL BE MEASURING A NUMBER OF BLOOD MARKERS OF HEART FUNCTION IN INDIVIDUALS WITH AND WITHOUT FRDA, AS A MEANS OF TRYING TO UNDERSTAND THE MECHANISMS WHICH MAY CONTRIBUTE TO THE HEART MUSCLE CHANGES. - CLINICAL TRIAL AT THE CHILDREN'S HOSPITAL OF PHILADELPHIA, PHILADELPHIA, PA(PRINCIPAL INEVSTIGATOR: DR. DAVID LYNCH)DR. LYNCH LED AN INVESTIGATOR INITIATED STUDY OPEN-LABEL, PILOT STUDY OF INTERFERON GAMMA (ACTIMMUNE) FOR THE TREATMENT OF FRIEDREICH'S ATAXIA IN 12 PATIENTS (CHILDREN) THAT WAS FUNDED BY FARA. THE STUDY IS NOW COMPLETE AND THE DATA IS BEING ANALYZED.ADDITIONALLY, FARA CONTINUES TO SUPPORT THE STANDARDIZED CHARACTERIZATION OF FRDA MOUSE MODELS AT THE JACKSON LABORATORY. IN ADDITION TO RESEARCH GRANTS FARA FUNDS THE ONGOING DEVELOPMENT OF VITAL CLINICAL RESEARCH INFRASTRUCTURE. CLINICAL RESEARCH INFRASTRUCTURE REFERS TO THE RESOURCES NEEDED TO FACILITATE ANY TYPE OF RESEARCH, INCLUDING CLINICAL TRIALS THAT INVOLVE PATIENTS. THESE RESOURCES CAN INCLUDE THINGS LIKE: PATIENT REGISTRY : THE ONLY INTERNATIONAL FRIEDREICH ATAXIA PATIENT REGISTRY WITH MORE THAN 2000 INDIVIDUALS ENROLLED. THIS REGISTRY CAPTURES DEMOGRAPHIC AND CLINICAL INFORMATION ON INDIVIDUALS WITH FA AND IS USED TO RECRUIT INDIVIDUALS FOR CLINICAL TRIALS WORLD WIDE. FARA PATIENT REGISTRY : WWW.CUREFA.ORG/REGISTRYCOLLABORATIVE CLINICAL RESEARCH NETWORK IN FA (CCRN IN FA): AN INTERNATIONAL NETWORK OF 10 CLINICAL RESEARCH CENTERS THAT WORK TOGETHER TO ADVANCE TREATMENTS AND CLINICAL CARE FOR INDIVIDUALS WITH FRIEDREICH'S ATAXIA. HAVING SUCH A NETWORK MEANS THAT THERE ARE TRAINED PHYSICIANS AND RESEARCH COORDINATORS READY TO DO CLINICAL RESEARCH STUDIES AND TRIALS. ALSO, THIS NETWORK IS BACKED BY A DATA COORDINATION CENTER THAT FACILITATES ALL ASPECTS OF DATA COLLECTION, DATABASE MANAGEMENT, AND STATISTICAL ANALYSIS OF STUDY DATA. TO LEARN MORE, VISIT WWW.CUREFA.ORG/NETWORK.HTML NATURAL HISTORY STUDY: LONGITUDINAL DATA (USUALLY ABOUT 10 YEARS) ON INDIVIDUALS WITH A DISEASE THAT DESCRIBES AND QUANTIFIES THE PROGRESSION OF THE DISEASE ALONG WITH THE SYMPTOMS AND MANIFESTATIONS OF THE DISEASE. NATURAL HISTORY CAN SOMETIMES SERVE AS THE BASIS FROM WHICH MEASUREMENTS CAN BE MADE TO DETERMINE EFFECTS OF NEW TREATMENTS, DRUGS OR INTERVENTIONS. 85036 RESEARCH CONFERENCES: FARA ORGANIZES AND SUPPORTS A NUMBER OF SCIENTIFIC CONFERENCES TO IMPROVE SHARING OF KNOWLEDGE, INSIGHTS AND ADVANCES AND BUILD COLLABORATIONS AND SYNERGISTIC CONNECTIONS BETWEEN FA RESEARCHERS. IN JUNE 2013, FARA ORGANIZED A TWO-DAY FRIEDREICH'S ATAXIA CLINICAL RESEARCH CONFERENCE THAT HOSTED 65 PARTICIPANTS INCLUDING CLINICIANS, SCIENTISTS, FARA SCIENTIFIC ADVISORS AND REPRESENTATIVES FROM GOVERNMENT REGULATORY AGENCIES WHO PRESENTED AND DISCUSSED THE FOLLOWING TOPICS ON FRIEDREICH'S ATAXIA: OVERVIEW OF CLINICAL RESEARCH IN FRIEDREICH'S ATAXIA, NEUROLOGICAL, METABOLIC AND CARDIAC CLINICAL ENDPOINTS AND BIOMARKERS, COLLABORATIVE CLINICAL RESEARCH NETWORK IN FA, CLINICAL MANAGEMENT GUIDELINES AND CLINICAL TRIAL DESIGN. IN OCTOBER 2013, FARA ORGANIZED A TWO-DAY FRIEDREICH'S ATAXIA GENE THERAPY MEETING TO HOST 17 PARTICIPANTS THAT INCLUDED CLINICIANS AND SCIENTISTS FOCUSING ON CREATING A CLINICAL DEVELOPMENT PLAN FOR GENE THERAPY IN FA DISCUSSING TRIAL DESIGN, PATIENT SELECTION CRITERIA, DELIVERY OF GENE THERAPY, OPTIMAL EFFICACY MEASURES AND WHAT IS NEEDED FOR APPROVAL FOR GOVERNMENT REGULATORY AGENCIES. THIS MEETING WAS CRITICAL TO SETTING THE FORWARD PATH OF THE GENE THERAPY PROGRAM IN FA. 168443 EDUCATION, AWARENESS & OUTREACH PROGRAMS: FRIEDREICH'S ATAXIA (FA) IS A RARE DISEASE; AFFECTING 1 IN 50,000 INDIVIDUALS. FARA IS DEDICATED TO RAISING AWARENESS FOR FA. FARA HAS UTILIZED BOTH TRADITIONAL AND SOCIAL MEDIA STRATEGIES TO BRING GREATER AWARENESS TO FA IN THE GENERAL PUBLIC AND TO ENGAGE AND EDUCATE THE FA COMMUNITY. FOR EXAMPLE, FARA CONDUCTED A SOCIAL MEDIA CAMPAIGN THAT ENCOURAGED COMMUNITY PARTICIPATION LEADING UP TO RARE DISEASE DAY AND FA AWARENESS DAY. FARA ALSO HOSTED WEBINARS TO INFORM THE COMMUNITY ABOUT THE LATEST DEVELOPMENTS IN FA RESEARCH AND SOME OF THE RESULTS OF FUNDED GRANTS.FARA CO-ORGANIZED TWO PATIENT SYMPOSIUMS WITH CLINICAL RESEARCH NETWORK SITES, CHILDREN'S HOSPITAL OF PHILADELPHIA (CHOP) AND UNIVERSITY OF SOUTH FLORIDA (USF). THE CHOP AND FARA TEAM LED AN ALL-DAY SYMPOSIUM AT IN KING OF PRUSSIA, PA ON OCTOBER 14TH, 2013. THERE WERE MORE THAN 150 PATIENT FAMILIES WHO PARTICIPATED. THE SYMPOSIUM PROVIDED:" CLINICAL UPDATES, INCLUDING CARDIOLOGY, NEUROLOGY, PHYSICAL THERAPY, AND PREGNANCY IN PATIENTS WITH FA;" UPDATES ON CURRENT RESEARCH AND CLINICAL TRIALS, INCLUDING GENE THERAPY, HDAC INHIBITORS AND" PANEL DISCUSSION ON ACCESSIBLE TRAVEL.THE SYMPOSIUM AT USF HAD ABOUT 200 ATTEND AND WAS STREAMED LIVE ON FACEBOOK AND THERE WERE SEVERAL HUNDRED MORE VIEWS. THE USF SYMPOSIUM FOCUSED ON CARDIAC ASPECTS OF FA, ADVANCES IN GENE THERAPY AND THE ANNOUNCEMENT OF A CLINICAL TRIAL OF DRUG TARGETING MITOCHONDRIAL DYSFUNCTION IN FA.ALSO, IN 2011 FARA LAUNCHED A NEW AMBASSADOR PROGRAM WHICH MENTORS AND ENCOURAGES INDIVIDUALS WITH FA TO DEVELOP AND SHARE THEIR PERSONAL STORY AND TO BEGIN GRASSROOTS OUTREACH TO RAISE AWARENESS. IN 2013, THE GROUP GREW TO 21 PARTICIPANTS. MANY OF THEM SHARED THEIR STORIES AND ON FARA'S NEWLY FORMED AMBASSADOR BLOG, HOSTED AND/ OR SPOKE AT FUNDRAISING EVENTS, AND RAISED AWARENESS AMONG COMMUNITY GROUPS AND/OR WITH THE MEDIA.ATAXIAN ATHLETE INITIATIVE (AAI)AAI IS A GRANT PROGRAM THAT PROVIDES, THROUGH A COMPETITIVE GRANT APPLICATION PROCESS, FUNDING FOR EQUIPMENT THAT ENABLES ELIGIBLE INDIVIDUALS WITH ATAXIA WHO HAVE DEMONSTRATED THE DESIRE TO STAY ACTIVE AND HEALTHY DESPITE THEIR DISABILITIES, TO START OR FURTHER DEVELOP THEIR ABILITIES IN ADAPTIVE CYCLING.TO QUALIFY, APPLICANTS MUST HAVE BEEN DIAGNOSED WITH SOME FORM OF ATAXIA. APPLICANTS ARE REQUIRED TO SUBMIT AN ESSAY SUMMARIZING THEIR EXPERIENCE WITH ATAXIA AND THEIR EFFORTS TO STAY ACTIVE. ALL APPLICATIONS ARE REVIEWED BY A PANEL OF DISABLED ATHLETES, PEOPLE WITH ATAXIA, AND OTHER REPRESENTATIVES OF THE ATAXIA COMMUNITY. GRANTS WILL WERE AWARDED TO A LIMITED NUMBER OF APPLICANTS BASED ON THE FOLLOWING:- DIAGNOSIS - APPLICANTS MUST HAVE BEEN DIAGNOSED WITH SOME FORM OFATAXIA TO QUALIFY;- APPLICANT'S PERCEIVED CHARACTER, DRIVE, AND COMMITMENT TO AN ACTIVELIFESTYLE;- FINANCIAL NEED AND- DETAILED RESEARCH AND KNOWLEDGE OF EQUIPMENT REQUESTED.IN 2013, FARA PARTIALLY FUNDED ADAPTIVE CYCLING EQUIPMENT TO SIX PEOPLE LIVING WITH ATAXIA (4 IN THE US AND 2 INTERNATIONALLY). THE TOTAL FOR EQUIPMENT FUNDED FOR THE DOMESTIC RECIPIENTS DID NOT EXCEED $5,000 NOR DID THE TOTAL FOR THE EQUIPMENT FUNDED FOR THE INTERNATIONAL RECIPIENTS. 3245983 1 1 0 1 0 0 0 0 0 0 1 0 1 0 0 1 1 0 0 0 1 1 0 0 1 0 0 1 0 1 0 0 0 0 0 0 0 0 1 0 0 0 0 0 0 0 0 1 2 0 1 6 1 0 0 0 0 0 1 1 0 0 0 0 X 16 15 0 0 0 0 0 0 0 1 1 0 0 1 1 1 1 1 1 1 1 0 AL AK AZ AR CA CO CT FL GA IL KS KY ME MD MS MI MN MS MO NH NJ NM NY NC ND OH OK OR PA RI SC TN UT VA WA WV WI MA X X X JENNIFER FARMER 4848796160 533 W UWCHLAN AVENUE DOWNINGTOWN PA 19335 RONALD BARTEK PRESIDENT/DIRECTOR 40.00 X X X 160000 0 10236 EDWARD RAMSEY CHAIRMAN 0.00 X X 0 0 0 MARILYN E DOWNING SECRETARY 0.00 X X 0 0 0 RUTH DEWITT TREASURER 0.00 X X 0 0 0 JENNIFER GOOD DIRECTOR 0.00 X 0 0 0 PAUL AVERY DIRECTOR 0.00 X 0 0 0 PETER CRISP DIRECTOR 0.00 X 0 0 0 THOMAS A DECOTIIS PHD DIRECTOR 0.00 X 0 0 0 DR EARL GILLER SCIENTIFIC DIRECTOR 0.00 X 0 0 0 DR HOLLY HEDRICK DIRECTOR 0.00 X 0 0 0 NICHOLAS A JOHNSON DIRECTOR 0.00 X 0 0 0 LAURA KALICK DIRECTOR 0.00 X 0 0 0 STEVE KLASKO DIRECTOR 0.00 X 0 0 0 BILL ALEFANTIS DIRECTOR 0.00 X 0 0 0 PETER PITTS DIRECTOR 0.00 X 0 0 0 BERNARD RAVINA SCIENTIFIC DIRECTOR 0.00 X 0 0 0 JENNIFER FARMER EXECUTIVE DIRECTOR 40.00 X 84460 0 7328 244460 0 17564 1 0 1 0 0 2608852 1243648 3852500 9955 9955 755622 705713 49909 49909 49909 608047 2608852 532567 75480 75480 3987844 0 0 135344 1728333 1728333 1077913 1077913 262024 201107 17691 43226 189953 95123 10816 84014 10500 10500 15141 9141 6000 36000 36000 23738 14244 8395 1099 22096 11048 2762 8286 71844 67046 3863 935 4828 3258 1570 903 903 4543 2480 2063 CREDIT CARD & BANK FEES 7130 202 1492 5436 BOOKS, SUBSCRIPTIONS & 6078 1153 1595 3330 BUSINESS REGISTRATION F 5404 1454 3950 MISCELLANEOUS 1615 1415 200 1409 1409 3469452 3245983 65130 158339 511067 1357092 726101 277365 77128 109720 8551 6719 4631 3682 1852 949 607771 836233 1100000 1100000 3032470 3688078 264360 381693 264360 381693 X 2572398 2972678 195712 333707 2768110 3306385 3032470 3688078 518392 19883 0 X X 0 1 X 1 0 X 3433440 2980429 2831171 3174652 4460547 16880239 3433440 2980429 2831171 3174652 4460547 16880239 996455 15883784 4041 57570 12704 21555 9955 105825 16986064 0.93510 0.90990 X RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED 0 0 5100 4000 9100 3992919 4002019 350101 87525 0 0 302630 350101 652731 979097 9100 9100 18200 75658 87525 163183 244775 200 3564 5100 5100 13964 4631 3682 949 949 INVESTMENT IN EDISON PHARMACEUTICALS, INC. 1100000 C 1100000 X 4540294 19883 532567 552450 3987844 0 3987844 4002019 532567 532567 3469452 0 3469452 PART X, LINE 2: FARA BELIEVES IT HAS APPROPRIATE SUPPORT FOR ANY TAX POSITIONS TAKEN AND, AS SUCH, DOES NOT HAVE ANY UNCERTAIN TAX POSITIONS THAT ARE MATERIAL TO THE FINANCIAL STATEMENTS. FARA'S FEDERAL RETURN OF ORGANIZATION EXEMPT FROM INCOME TAX (FORM 990) FOR TAX YEARS 2010, 2011 AND 2012 IS SUBJECT TO EXAMINATION BY THE IRS, GENERALLY FOR THREE YEARS AFTER THE TAX RETURNS WERE FILED. PART XI, LINE 2D - OTHER ADJUSTMENTS: SPECIAL EVENTS EXPENSE PART XII, LINE 2D - OTHER ADJUSTMENTS: SPECIAL EVENTS EXPENSE 1 EUROPE 0 0 GRANTS TO RECIPIENTS LOCATED IN REGION RESEARCH AND GRANT PROGRAM 722073 EAST ASIA AND THE PACIFIC 0 0 GRANTS TO RECIPIENTS LOCATED IN REGION RESEARCH AND GRANT PROGRAM 355840 0 0 0 0 0 0 1077913 0 1077913 EUROPE MEDICAL RESEARCH 158600 WIRE EUROPE MEDICAL RESEARCH 100000 WIRE EAST ASIA AND THE PACIFIC MEDICAL RESEARCH 83519 WIRE EUROPE MEDICAL RESEARCH 70000 WIRE EUROPE MEDICAL RESEARCH 113473 WIRE EAST ASIA AND THE PACIFIC MEDICAL RESEARCH 121508 WIRE EUROPE MEDICAL RESEARCH 50000 WIRE EUROPE MEDICAL RESEARCH 230000 WIRE EAST ASIA AND THE PACIFIC MEDICAL RESEARCH 150813 WIRE 0 0 0 0 0 0 FARA ENERGY BALL 1580782 1086378 494404 223806 RIDE ATAXIA PHILADELPHIA 253058 241183 11875 32488 1383059 1281291 101768 276273 3216899 2608852 608047 532567 532567 75480 FELICIA DEROSA 533 W UWCHLAN AVENUE DOWNINGTOWN PA 19335 1 UNIVERSITY OF ROCHESTER 515 HYLAN BUILDING RC BOX 270140 ROCHESTER NY 14627 160743209 501(C)(3) 205919 MEDICAL RESEARCH UNIVERSITY OF SOUTH FLORIDA FOUNDATION 4202 EAST FOWLER AVENUE TAMPA FL 33620 590879015 501(C)(3) 150000 MEDICAL RESEARCH CHILDREN'S HOSPITAL OF PHILADELPHIA 34TH ST CIVIC CENTER BLVD PHILADELPHIA PA 19104 231352166 501(C)(3) 304310 MEDICAL RESEARCH OHIO STATE UNIVERSITY 333 W 10TH AVE COLUMBUS OH 43210 316025986 501(C)(3) 4800 MEDICAL RESEARCH SCRIPPS RESEARCH INSTITUTE 10550 NORTH TORREY PINES ROAD OSP-TPC7 LA JOLLA CA 92037 330435954 501(C)(3) 25385 MEDICAL RESEARCH THE JACKSON LABORATORY 600 MAIN STREET BAR HARBOR ME 04609 010211513 501(C)(3) 37189 MEDICAL RESEARCH THE REGENTS OF THE UNIVERSITY OF CALIFORNIA LOS ANGELES 10920 WILSHIRE BLVD 5TH FLOOR LOS ANGELES CA 90024 956006143 501(C)(3) 150082 MEDICAL RESEARCH EMORY UNIVERSITY 201 DOWMAN DRIVE ATLANTA GA 30322 580566256 501(C)(3) 21100 MEDICAL RESEARCH UNIVERSITY OF CHICAGO 5801 SOUTH ELLIS AVENUE CHICAGO IL 60637 362177139 501(C)(3) 2500 MEDICAL RESEARCH UNIVERSITY OF IOWA DEPT OF PEDIATRICS 200 HAWKINS DRIVE IOWA CITY IA 52242 426004813 501(C)(3) 3200 MEDICAL RESEARCH THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA 3451 WALNUT STREET RM P 221 FRANKLIN BLDG PHILADELPHIA PA 19104 231352685 501(C)(3) 61441 MEDICAL RESEARCH INDIANA UNIVERSITY 1044 WEST WALNUT INDIANAPOLIS IN 46202 356001673 501(C)(3) 100000 MEDICAL RESEARCH REPLIGEN 41 SEYON ST WALTHAM MA 02453 042729386 68405 MEDICAL RESEARCH UNIVERSITY OF TEXAS MD ANDERSON 1515 HOLCOMBE BLVD UNIT 1676 HOUSTON TX 77030 746001118 501(C)(3) 30290 MEDICAL RESEARCH UNIVERSITY OF FLORIDA - DEPT OF NEUROLOGY L3-100 MCKNIGHT BRAIN INSTITUTE NEWELL DRIVE GAINESVILLE FL 32611 596002052 501(C)(3) 7400 MEDICAL RESEARCH ALBANY RESEARCH INSTITUTE INC 113 HOLLAND AVE ALBANY NY 12208 141716021 501(C)(3) 90292 MEDICAL RESEARCH DUKE UNIVERSITY 134 CHAPEL DRIVE DURHAM NC 27708 560532129 501(C)(3) 100000 MEDICAL RESEARCH FEDERATION OF AMERICAN SOCIETIES FOR EXPERIMENTAL BIOLOGY 9650 ROCKVILLE PIKE BETHESDA MD 20814 520700497 501(C)(3) 5000 MEDICAL RESEARCH LSU HEALTH SCIENCE CENTER 433 BOLIVAR ST NEW ORLEANS LA 70112 726087770 501(C)(3) 89335 MEDICAL RESEARCH REGENTS OF THE UNIVERSITY OF MINNESOTA 200 SE OAK ST 600 MINNEAPOLIS MN 55455 416007513 501(C)(3) 128338 MEDICAL RESEARCH RETROTOPE 12133 FOOTHILL LAN LOS ALTOS HILL CA 94022 204958003 44250 MEDICAL RESEARCH WEILL MEDICAL COLLEGE OF CORNELL UNIVERSITY 445 E 69TH ST NEW YORK NY 10021 131623978 501(C)(3) 95497 MEDICAL RESEARCH FES13 CONFERENCE 15600 HAMPTON STREET ANNEX RM 208 COLUMBIA SC 29208 576001153 501(C)(3) 3600 MEDICAL RESEARCH PART I, LINE 2: GRANTS ARE REVIEWED BY INDEPENDENT SCIENTIFIC ADVISORS AND APPROVED BY BOARD. X 0 0 0 0 0 0 0 0 0 RONALD BARTEK PRESIDENT/DIRECTOR 160000 0 0 0 0 0 0 0 10236 0 170236 0 0 0 X 3 44818 MARKET PRICE 0 0 0 FORM 990, PART III, LINE 4A CONTINUED CLINICAL OUTCOME MEASURES : FUNCTIONAL PERFORMANCE TESTS (E.G., TIMED PEGBOARD OR WALK TESTS, VISION, HEARING OR SPEECH TESTS) THAT QUANTIFY HOW MUCH CHANGE TAKES PLACE IN A SPECIFIC AMOUNT OF TIME AND ARE USED IN CLINICAL TRIALS TO MEASURE WHETHER A DRUG IS ALTERING THE COURSE OF THE DISEASE. BIOMARKERS: ANYTHING THAT CAN BE USED AS AN INDICATOR OF A PARTICULAR DISEASE STATE - USUALLY PROTEINS, ENZYMES, GENETIC VARIANTS, IMAGING (MRI, CT OR PET SCANS). BIOMARKERS CAN BE USED TO ASSESS RISK OF DISEASE, DIAGNOSIS, OR OUTCOMES. USE OF BIOMARKERS IN DRUG DEVELOPMENT IS OF GREAT INTEREST BECAUSE BIOMARKERS CAN PROVIDE EVIDENCE OF BIOLOGICAL ACTIVITY, POTENTIALLY DEMONSTRATING THERAPEUTIC BENEFIT MORE QUICKLY THAN TRADITIONAL OUTCOME MEASURES. BIOREPOSITORY: A REPOSITORY OR BANK OF STORED BIOLOGICAL MATERIALS SUCH AS BLOOD SAMPLES, DNA, ORGANS, AND TISSUES (SUCH AS SKIN, MUSCLE, HEART) THAT CAN BE USED FOR RESEARCH. THROUGH THE CCRN IN FA WE HAVE COLLECTED TEN YEARS OF NATURAL HISTORY DATA (ONGOING) IN MORE THAN 700 INDIVIDUALS WITH FA, VALIDATED CLINICAL OUTCOME MEASURES AND THE FARS SCALE, STUDIED SPEECH, VISION AND HEARING, LAUNCHED BIOMARKER STUDIES, ESTABLISHED DNA AND RNA REPOSITORIES, AND PROVIDED MANY BLOOD SAMPLES TO RESEARCHERS AROUND THE WORLD. THE CCRN IN FA INVESTIGATORS HAVE BEEN INVOLVED IN MULTIPLE CLINICAL TRIALS INCLUDING A FEW THAT WERE DESIGNED AND CONDUCTED SOLELY THROUGH NETWORK SITES. FARA CONTRIBUTED MORE THAN $550,000 IN 2013 TO CLINICAL RESEARCH INFRASTRUCTURE IN DIRECT FUNDING TO THE CCRN IN FA CENTERS AND RELATED CLINICAL RESEARCH ACTIVITIES. TITLES AND SUMMARIES OF MOST OF THE PROJECTS PRESENTLY FUNDED BY FARA ARE AVAILABLE AT: WWW.CUREFA.ORG/RPMP/PUBLIC/PGGRANTLIST.ASPX AND COMPLETE LISTINGS OF GRANTS AWARDED BY YEAR CAN BE ACCESSED AT: WWW.CUREFA.ORG/GRANTS-AWARDED.HTML FORM 990, PART VI, SECTION B, LINE 11 COPIES OF 990 ARE DISTRIBUTED TO BOARD MEMBERS FOR REVIEW AND APPROVAL PRIOR TO FILING. FORM 990, PART VI, SECTION B, LINE 12C ALL NEW AND EXISTING BOARD MEMBERS REQUIRED TO ANNUALLY COMPLETE A CONFLICT OF INTEREST STATEMENT FORM 990, PART VI, SECTION B, LINE 15 EVALUATION AND COMPENSATION COMMITTEE PERFORMS EMPLOYEE EVALUATIONS AND DETERMINES SALARY INCREASES ON A YEARLY BASIS FOR ALL EMPLOYEES. COMMITTEE EXAMINES BENCHMARK DATA IN DETERMINING SALARIES FOR PRESIDENT AND EXECUTIVE DIRECTOR. FORM 990, PART VI, SECTION C, LINE 18 FORM 990 IS AVAILABLE ON THE ORGANIZATION'S WEBSITE AS WELL AS GUIDESTAR.ORG AND CHARITYNAVIGATOR.ORG FORM 990, PART VI, SECTION C, LINE 19 FINANCIAL STATEMENTS ARE AVAILABLE ON THE ORGANIZATION'S WEBSITE. THE GOVERNING DOCUMENTS, CONFLICT OF INTEREST POLICY AND FORM 1023 ARE AVAILABLE UPON REQUEST. FORM 990, PART VI LINE 17 THE ORGANIZATION IS ALSO REGISTERED TO SOLICIT CONTRIBUTIONS IN THE STATES LISTED. FORM 990, PART XII, LINE 2C FARA'S AUDIT COMMITTEE CONSISTS OF THE EXECUTIVE COMMITTEE, EXECUTIVE DIRECTOR AND ONE AT-LARGE BOARD MEMBER. EACH YEAR THE AUDIT COMMITTEE SEEKS THE SERVICES OF AN OUTSIDE ACCOUNTING FIRM AND CONTRACTS FOR A FULL AUDIT, PREPARATION OF FINANCIAL STATEMENTS AND FILING OF THE 990. THE AUDIT COMMITTEE IS RESPONSIBLE FOR REVIEWING RECOMMENDATIONS FROM THE AUDIT AND PROPOSING NEW POLICIES AND PROCEDURES AS NECESSARY. THE AUDIT COMMITTEE ALSO PARTICIPATES IN DETAILED REVIEW OF FINANCIAL STATEMENTS AND 990 PRIOR TO SHARING WITH THE FULL BOARD FOR A VOTE. FARA'S BOARD OF DIRECTORS RECEIVES THE FINANCIAL STATEMENTS AND 990 FOR REVIEW AND VOTES TO APPROVE PRIOR TO PUBLIC FILING.